A new strategy aims to improve gene therapy for Pompe disease by optimizing both the genetic component that restores the function of a deficient lysosomal enzyme and the vector that delivers it to the target tissue while avoiding the liver. The findings suggest that combining an optimized transgene with a targeted capsid could significantly enhance the effectiveness of gene therapy for Pompe disease.
Showing a significant efficacy signal in a phase II trial, Relay Therapeutics Inc.’s zovegalisib (RLY-2608) achieved a 60% volumetric response in patients with PIK3CA-driven vascular anomalies (VAs). The isoform-selective PI3Ka inhibitor is in late-stage clinical trials with various combinations for P13Ka-mutated, HR+/HER2- advanced breast cancer, with VAs representing a second indication for which Leerink Partners analyst Andrew Berens forecasts $2.8 billion in peak revenues.
How the U.S. FDA might view the latest Duchenne muscular dystrophy (DMD) phase III data with gene therapy RGX-202 became the question for Regenxbio Inc., shares of which (NASDAQ:RGNX) closed May 14 at $6.25, down 38%, or $3.80, after the results were made public.
